An experimental rare disease drug from Sanofi succeeded against one so-called lysosomal storage disorder but failed against another, the French pharmaceutical company said Monday.
According to Sanofi, the drug, dubbed venglustat, missed its primary objective in a Phase 3 study testing it against Fabry disease. However, in another study in a form of Gaucher disease, the drug met its main goal and three out of four key secondary endpoints. Sanofi didn’t provide details — they’ll be shared at medical meeting this week — but said it intends to submit the Gaucher results to global regulatory authorities.
Sanofi has viewed venglustat as a key part of its future for more than a decade. The company inherited the medication as part of an acquisition of the rare disease drug specialist Genzyme and, since then, has tested it against a variety of conditions. It’s meant to lower the production of a kind of toxic substrate, in turn reducing the buildup of potentially dangerous materials in cells — such as a certain fatty substance in Gaucher disease.
The drug’s development has been marred by clinical setbacks, however. Prior to Monday’s announcement, venglustat failed studies in Parkinson’s disease, a rare kidney condition, as well as another lysosomal storage disorder known as GM2 gangliosidosis.
On Monday, Fabry was added to the list. In a Phase 3 trial, venglustat wasn’t associated with a meaningful improvement in patient-reported pain scores, compared to a placebo. Sanofi said additional analyses are ongoing and more information will be shared at a future medical meeting. A second Phase 3 study of venglustat in Fabry disease is ongoing, Sanofi said.
The drug did, though, succeed in a study of people with the “Type 3” form of Gaucher, which affects brain function and can lead to progressive neurological impairment. While people with Gaucher can get an enzyme replacement therapy — Sanofi sells one called Cerezyme — those treatments don’t help with the disease’s effects on the brain.
Sanofi tested venglustat against an enzyme replacement therapy in the study. It found that, after a year of treatment, those who received its therapy had statistically significant improvements, comparatively, on two tests evaluating neurological symptoms. The drug was “well tolerated,” though instances of nausea, diarrhea and spleen enlargement were higher among recipients, Sanofi said.
"What excites us most is the potential to address critical unmet medical needs,” said Houman Ashrafian, Sanofi’s head of research and development, in the statement. “A daily pill could make a serious difference for Gaucher patients facing neurological challenges.”
The venglustat readouts come after a few recent pipeline setbacks for Sanofi. An eczema drug that’s currently one of the company’s top prospects produced results that fell short of expectations in a late stage trial. An important multiple sclerosis drug also has an uncertain future.
